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Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

22 juillet 2017 mis à jour par: Novartis Pharmaceuticals

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

173

Phase

  • Phase 2

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

  • Allemagne
      • Berlin, Allemagne, 13353
        • Novartis Investigative Site
      • Dresden, Allemagne, 01307
        • Novartis Investigative Site
      • Goslar, Allemagne, 38642
        • Novartis Investigative Site
      • Hannover, Allemagne, 30170
        • Novartis Investigative Site
      • Leipzig, Allemagne, 04103
        • Novartis Investigative Site
      • Potsdam, Allemagne, 14467
        • Novartis Investigative Site
    • Baden-Württemberg
      • Mannheim, Baden-Württemberg, Allemagne, 68305
        • Novartis Investigative Site
  • Arabie Saoudite
      • Dammam, Arabie Saoudite, 15215
        • Novartis Investigative Site
      • Dammam, Arabie Saoudite, 40145
        • Novartis Investigative Site
      • Jeddah, Arabie Saoudite, 21589
        • Novartis Investigative Site
      • Riyadh, Arabie Saoudite, 11472
        • Novartis Investigative Site
  • Argentine
      • Buenos aires, Argentine, C1221ADC
        • Novartis Investigative Site
  • Emirats Arabes Unis
      • Al Ain - Abu Dhabi, Emirats Arabes Unis
        • Novartis Investigative Site
      • Dubai, Emirats Arabes Unis, 9115
        • Novartis Investigative Site
  • Espagne
      • Barcelona, Espagne, 08041
        • Novartis Investigative Site
      • Madrid, Espagne, 28033
        • Novartis Investigative Site
  • France
      • Lille cedex, France, 59020
        • Novartis Investigative Site
      • Paris, France, 75010
        • Novartis Investigative Site
  • Fédération Russe
    • Russia
      • Moskow, Russia, Fédération Russe, 117198
        • Novartis Investigative Site
  • Grèce
    • GR
      • Athens, GR, Grèce, GR-115 27
        • Novartis Investigative Site
      • Patra - RIO, GR, Grèce, 265 04
        • Novartis Investigative Site
      • Thessaloniki, GR, Grèce, 546 42
        • Novartis Investigative Site
  • Italie
      • Napoli, Italie, 80138
        • Novartis Investigative Site
    • BR
      • Brindisi, BR, Italie, 72100
        • Novartis Investigative Site
    • CT
      • Catania, CT, Italie, 95125
        • Novartis Investigative Site
    • FE
      • Cona, FE, Italie, 44100
        • Novartis Investigative Site
    • GE
      • Genova, GE, Italie, 16132
        • Novartis Investigative Site
      • Genova, GE, Italie, 16128
        • Novartis Investigative Site
    • ITA
      • Cagliari, ITA, Italie, 09121
        • Novartis Investigative Site
    • LE
      • Lecce, LE, Italie, 73100
        • Novartis Investigative Site
    • MI
      • Milano, MI, Italie, 20162
        • Novartis Investigative Site
      • Milano, MI, Italie, 20122
        • Novartis Investigative Site
    • PA
      • Palermo, PA, Italie, 90127
        • Novartis Investigative Site
      • Palermo, PA, Italie, 90146
        • Novartis Investigative Site
    • RC
      • Reggio Calabria, RC, Italie, 89100
        • Novartis Investigative Site
    • VR
      • Verona, VR, Italie, 37126
        • Novartis Investigative Site
  • L'Autriche
      • Linz, L'Autriche, A-4010
        • Novartis Investigative Site
      • Wien, L'Autriche, 1140
        • Novartis Investigative Site
  • Liban
    • Beirut
      • Hazmiyeh, Beirut, Liban, PO BOX 213
        • Novartis Investigative Site
  • Malaisie
      • Kuala Lumpur, Malaisie, 50589
        • Novartis Investigative Site
      • Pulau Pinang, Malaisie, 10990
        • Novartis Investigative Site
  • Mexique
    • Distrito Federal
      • Mexico, Distrito Federal, Mexique, 06726
        • Novartis Investigative Site
  • Royaume-Uni
      • London, Royaume-Uni, N19 5NF
        • Novartis Investigative Site
      • London, Royaume-Uni, NW1 2PJ
        • Novartis Investigative Site
  • Thaïlande
      • Bangkok, Thaïlande, 10700
        • Novartis Investigative Site
      • Bangkok, Thaïlande, 10400
        • Novartis Investigative Site
  • États-Unis
    • California
      • Orange, California, États-Unis, 92868-3874
        • Children's Hospital of Orange County Onc Dept
    • Illinois
      • Chicago, Illinois, États-Unis, 60611
        • Lurie Children's Hospital of Chicago Onc Dept
    • Massachusetts
      • Boston, Massachusetts, États-Unis, 02115
        • Children's Hospital Boston Department of Hematology
    • New York
      • New York, New York, États-Unis, 10021
        • Weill Cornell Medical College-Cornell University Onc Dept
    • Pennsylvania
      • Philadelphia, Pennsylvania, États-Unis, 19104-4399
        • Children's Hospital of Philadelphia Onc. Dept

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

10 ans et plus (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

Key Inclusion Criteria:

  • Male and female patients aged ≥ 10 years
  • Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
  • History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
  • Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).

Key Exclusion Criteria:

  • Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
  • Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
  • ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
  • Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
  • Liver disease with severity of Child-Pugh Class B or C

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Comparateur actif: Deferasirox dispersible tablet (DFX-DT)
Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose.
Médicament: Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Autres noms:
  • ICL670, DT (dispersible tablet)
Expérimental: Deferasirox film-coated tablet (DFX-FCT)
Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose
Médicament: Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Autres noms:
  • ICL670, FCT (film-coated tablet)

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Overall Safety as Measured by Frequency of Adverse Events
Délai: 28 weeks
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
28 weeks
Overall Safety as Measured by Changes in Laboratory Values From Baseline
Délai: baseline (BL), 30 weeks
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
baseline (BL), 30 weeks

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Frequency of Selected Gastro-intestinal (GI) Adverse Events
Délai: 28 weeks
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
Délai: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
Délai: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of "no aftertaste". The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
Délai: weeks -1, 4, 8, 12, 16, 20, 24
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
Délai: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
Délai: weeks 1, 4, 8, 12, 16, 20, 24
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100.
weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Délai: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess AUClast.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Délai: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Cmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Délai: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Tmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
Délai: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT.
Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Novartis Pharmaceuticals

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

8 juillet 2014

Achèvement primaire (Réel)

24 février 2016

Achèvement de l'étude (Réel)

24 février 2016

Dates d'inscription aux études

Première soumission

26 avril 2014

Première soumission répondant aux critères de contrôle qualité

26 avril 2014

Première publication (Estimation)

29 avril 2014

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

25 juillet 2017

Dernière mise à jour soumise répondant aux critères de contrôle qualité

22 juillet 2017

Dernière vérification

1 juillet 2017

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • CICL670F2201

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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